CUDC-907 for Pediatric Cancer

Recruiting at 3 trial locations

Overseen By David S Shulman, MD

Age: < 65

Sex: Any

Trial Phase: Phase 1

Sponsor: Dana-Farber Cancer Institute

Must not be taking: Corticosteroids, NSAIDs, Anticoagulants, Heparins

Disqualifiers: Pregnancy, Breastfeeding, Diabetes, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: Up to 2 years

26 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial is testing a new drug called CUDC-907 to see if it can help children with certain cancers that haven't responded to other treatments. The drug works by blocking proteins that help cancer cells grow. The main goal is to find the safest dose for children and see if the drug has any benefits.

Will I have to stop taking my current medications?

The trial protocol does not specify if you must stop all current medications, but you cannot take certain drugs like systemic corticosteroids (unless for specific conditions), non-steroidal anti-inflammatory drugs, oral anticoagulants, and therapeutic heparins. It's best to discuss your current medications with the study team to see if they are allowed.

Is CUDC-907 safe for use in children with cancer?

There is no specific safety data available for CUDC-907 in children with cancer from the provided research articles. However, the general safety of anticancer drugs in children often involves challenges due to off-label use and the potential for unknown adverse reactions. ^{1 2 3 4 5}

What makes the drug CUDC-907 unique for treating pediatric cancer?

CUDC-907 is unique because it is a first-in-class, oral drug that targets both PI3K and HDAC enzymes, which are involved in cancer cell growth and survival. This dual action is different from many existing treatments that typically target only one pathway, potentially making CUDC-907 more effective in certain cancers. ^{6 7 8 9 10}

Who Is on the Research Team?

David S Shulman, MD

Principal Investigator

Dana-Farber Cancer Institute

Are You a Good Fit for This Trial?

This trial is for children and young adults aged 1 to 21 with relapsed or refractory solid tumors, CNS tumors, or lymphoma. They must have a performance status of at least 50%, measurable disease, normal organ function, be able to swallow medication without chewing, and not be pregnant or breastfeeding. Participants need stable neurological conditions and can't be on certain medications like corticosteroids (unless for specific diseases), NSAIDs, anticoagulants, or have uncontrolled illnesses.

Inclusion Criteria

Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy

Patients must have normal organ function as defined

Patients must be able to swallow either intact capsules or mini-tabs without chewing

Exclusion Criteria

Patients must not be receiving non-steroidal anti-inflammatory drugs, oral anticoagulants, and therapeutic heparins

Pregnant participants will not be entered on this study

Breastfeeding mothers are not eligible

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive CUDC-907 orally once daily for 5 consecutive days per week followed by two days without dosing. Dose escalation follows a standard 3+3 design.

Up to 2 years

Weekly visits for dose administration and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• CUDC-907

Trial Overview The study is testing CUDC-907's effectiveness in treating pediatric patients who haven't responded well to standard treatments for various types of cancer including neuroblastoma and brain tumors. It aims to find out if this new drug can help where other treatments haven't worked.

How Is the Trial Designed?

Group I: CUDC-907 Experimental Treatment 1 Intervention

* CUDC-907 orally administered * CUDC-907 once daily for 5 consecutive days per week followed by two days without dosing * Dose level assigned at registration * Pre-dose pharmacokinetic blood sample will be collected * Dose escalation will follow a standard 3+3 design

Who Is Running the Clinical Trial?

Dana-Farber Cancer Institute

Lead Sponsor

Trials

1,128

Recruited

382,000+

Curis, Inc.

Industry Sponsor

Trials

17

Recruited

1,100+

Published Research Related to This Trial

Out of 236 Individual Case Safety Reports (ICSRs) analyzed, only 18 (7.6%) were classified as off-label cases, indicating a low incidence of off-label use of antineoplastic drugs in pediatric patients, primarily related to therapeutic indications.

Among the off-label cases, 29 adverse drug reactions (ADRs) were reported, with common reactions including diarrhea and neutropenia, suggesting that while off-label use is limited, it can still lead to significant safety concerns.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety of Anticancer Agents Used in Children: A Focus on Their Off-Label Use Through Data From the Spontaneous Reporting System. Mascolo, A., Scavone, C., Bertini, M., et al.[2022]

Citations

1. United States pubmed.ncbi.nlm.nih.gov

Chemotherapy-related toxicity in infants treated according to the Second National Wilms' Tumor Study. [2017]

2. United States pubmed.ncbi.nlm.nih.gov

Subjective Toxicity Profiles of Children in Treatment for Cancer: A New Guide to Supportive Care? [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Opportunities and Challenges in Drug Development for Pediatric Cancers. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

Cancer drugs approved for use in children: Impact of legislative initiatives and future opportunities. [2020]

5. Switzerland pubmed.ncbi.nlm.nih.gov

Safety of Anticancer Agents Used in Children: A Focus on Their Off-Label Use Through Data From the Spontaneous Reporting System. [2022]

6. Italy pubmed.ncbi.nlm.nih.gov

CUDC-907 in relapsed/refractory diffuse large B-cell lymphoma, including patients with MYC-alterations: results from an expanded phase I trial. [2019]

7. Switzerland pubmed.ncbi.nlm.nih.gov

Novel Mechanistic Insight into the Anticancer Activity of Cucurbitacin D against Pancreatic Cancer (Cuc D Attenuates Pancreatic Cancer). [2022]

8. Netherlands pubmed.ncbi.nlm.nih.gov

CUDC-907 enhances TRAIL-induced apoptosis through upregulation of DR5 in breast cancer cells. [2020]

9. United States pubmed.ncbi.nlm.nih.gov

CUDC-907 displays potent antitumor activity against human pancreatic adenocarcinoma in vitro and in vivo through inhibition of HDAC6 to downregulate c-Myc expression. [2023]

10. England pubmed.ncbi.nlm.nih.gov

Safety, tolerability, and preliminary activity of CUDC-907, a first-in-class, oral, dual inhibitor of HDAC and PI3K, in patients with relapsed or refractory lymphoma or multiple myeloma: an open-label, dose-escalation, phase 1 trial. [2019]